Any drugmaker knows that no matter what a drug's sticker price, its sales can only go as far as its patient pool will take it. So for Vertex ($VRTX), whose cystic fibrosis med Kalydeco had reached nearly all eligible patients in the U.S. and Europe, a new FDA approval to treat more CF sufferers is pretty significant.
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| Courtesy of Vertex |
Orphan drug Kalydeco, originally approved just over two years ago for CF patients with at least one copy of the G551D mutation, snagged an FDA thumbs-up Friday for 8 additional mutations. The green light widens Kalydeco's eligible patient population by 150 in the U.S., which, considering Kalydeco's $300,000 price tag, spells more than $45 million in potential new revenue, according to the Boston Business Journal.
The sNDA approval follows a Phase III study of 39 people with 9 different mutations. Patients with the G178R, S549N, S549R, G551S, G1244E, S1251N and S1255P mutations--those for which Kalydeco won the FDA's blessing Friday--showed statistically significant improvements in lung function with a safety profile similar to prior studies, the company said. Kalydeco failed at treating only one mutation, G970R, which affects approximately 10 people worldwide.
The FDA's blessing bodes well for Vertex as it waits for decisions from Europe, Canada and Australia concerning the same mutations. Europe and Australia are home to approximately 250 potentially eligible patients, so approvals there would help Vertex hit its 2014 revenue predictions of between $570 million and $600 million; the company expects Kalydeco to generate $470 to $500 million of that.
"We believe that Kalydeco has the potential to help more people with CF, and today's approval is an important step toward that goal," Robert Kauffman, Vertex SVP and co-Chief Medical Officer, said in a Friday statement. "As we progress over the coming year, we look forward to data from multiple other ongoing studies that are designed to evaluate whether additional people with CF may benefit from Kalydeco."
A couple such studies, Traffic and Transport, made news a few days back when Vertex swapped their primary and secondary endpoints. As TheStreet's Adam Feuerstein reports, the primary endpoint of both studies, which test the combination of Kalydeco and VX-809 in CF patients with the F508del homozygous mutation, is now the absolute change in lung function, as opposed to relative change in lung function--a change spokesman Zach Barber called "procedural." Results could deliver a game-changing $6 billion in sales, Feuerstein notes.
But Vertex has seen some failures, too. Last December, Kalydeco flunked a Phase III trial on patients with the R117H mutation, failing to reach statistical significance in improving lung function compared with placebo. It did, however, hit primary and secondary endpoints for those 18 and older, a 300-patient subset in the U.S.
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